8 Best Gene-Editing Stocks to Buy

2. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX)

Number of Hedge Fund Holders: 68 

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is a biotechnology company that develops transformative medicines for serious diseases, with a strong focus on gene editing, particularly in collaboration with CRISPR Therapeutics. The company specializes in treatments for genetic diseases, especially cystic fibrosis, and generates revenue by selling medications to patients with rare genetic conditions. Recently, the company has expanded its focus to include sickle cell disease and beta-thalassemia.

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) has partnered with CRISPR Therapeutics to develop CASGEVY (exagamglogene autotemcel), a gene-edited cell therapy for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). This treatment involves editing the BCL11A gene in a patient’s stem cells to increase the production of fetal hemoglobin, which helps manage these inherited blood disorders.

The company’s Q4 2024 financial results highlight continued growth, with a 16% increase in revenue from the same period in 2023, reaching $2.91 billion. Full-year 2024 revenue reached $11 billion, exceeding expectations. The growth was driven by strong sales of CF treatments and the launch of CASGEVY. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is also expanding its reach with new therapies like ALYFTREK for CF and JOURNAVX for acute pain, solidifying its place among the best gene editing stocks.

With a broad total addressable market (TAM) covering diseases like CF, SCD, TDT, acute pain, type 1 diabetes, and renal diseases, the company aims to treat millions of patients worldwide. The business is focused on growing its market presence and advancing its innovative therapies.